Duchenne muscular dystrophy

Moorfields research provides hope for choroideremia curePreliminary research has shown that ataluren is effective in overriding the nonsense mutation in choroideremia patients, however, further research is needed.
Elevated CPK: No short cut to muscular dystrophy diagnosisA pediatrician with special training in neuromuscular disorders cautions that nothing replaces the traditional workup—a complete history and physical—for diagnosing neuromuscular complaints.
Oral specialty drug options on the horizon
Oral specialty drug options on the horizonAs more specialty drugs are approved, cost control strategies are key.
Controversy surrounds Exondys 51 approval: What to knowNew medicine for Duchenne muscular dystrophy raises questions. Here’s whose covering it and why.
Incremental clinical advances for Duchenne Muscular DystrophyJust last year, the FDA approved Exondys 51 (eteplirsen) for DMD, over the recommendations of FDA scientists and an external scientific advisory committee. Read more.
9 Newly Approved Novel Drugs
9 Newly Approved Novel DrugsIt's still early in 2017, but the FDA has been approving drugs left and right. Here's what you need to know.
Muscular dystrophy drug launch halts after pricing concernsMarathon Pharmaceuticals is temporarily pausing the launch of its drug to treat a rare form of muscular dystrophy (MD) after concerns over its $89,000 price tag.
FDA approves first drug for Duchenne muscular dystrophyEven though an FDA panel recommended against approving a new drug to treat Duchenne muscular dystrophy in April, the agency granted accelerated approval to eteplirsen injection (Exondys 51, Sarepta Therapeutics) in mid-September.
Three drug trends impacting specialty pharmacyAMCP session, “Specialty Pharmaceutical in Development,” highlighted critical trends that healthcare executives should be watching
FDA drug approvals-April 2015FDA actions in brief, priority review, breakthrough therapy designation, fast-track designations, orphan drug designations, first-time generic approvals